In short
Pralsetinib is an emerging targeted therapy being investigated in clinical trials for the treatment of various cancers with RET gene alterations. This article summarizes key information about ongoing clinical trials evaluating pralsetinib's safety and efficacy in conditions like non-small cell lung cancer, thyroid cancer, and other RET-altered solid tumors. The trials aim to assess pralsetinib as a potential treatment option for patients with limited alternatives.
At a glance
- Drug Name
- Pralsetinib (BLU-667)
- Target Conditions
- RET fusion-positive NSCLC, Medullary Thyroid Cancer, RET-altered solid tumors
- Administration
- Oral, 400 mg once daily in 28-day cycles
- Trial Phases
- Phase 1/2, Phase 2, Phase 3
- Key Outcomes Measured
- Objective response rate, progression-free survival, overall survival, safety profile
- Patient Populations
- Adults and select adolescents with RET-altered cancers, including treatment-naive and previously treated patients
- Notable Trial Designs
- Basket trials for multiple tumor types, neoadjuvant/adjuvant studies, comparisons to standard of care
What is Pralsetinib?
Pralsetinib is a targeted cancer therapy drug. It is also known by the brand names Gavreto in the United States and BLU-667. Pralsetinib belongs to a class of medications called tyrosine kinase inhibitors (TKIs). These drugs work by targeting specific proteins in cancer cells that help them grow and spread.
How Pralsetinib Works
Pralsetinib is designed to target a specific genetic alteration called RET fusion or mutation. RET stands for "rearranged during transfection," which refers to a gene that can become altered in some cancers. When the RET gene fuses with another gene or develops mutations, it can lead to uncontrolled cell growth and cancer.
Pralsetinib works by blocking the activity of the altered RET protein, which can help slow or stop the growth of cancer cells with these specific genetic changes. This targeted approach means that pralsetinib may be effective against cancers with RET alterations, regardless of where in the body the cancer originated.
Conditions Treated with Pralsetinib
Pralsetinib is being studied and used to treat several types of cancer that have RET gene alterations, including:
- Non-Small Cell Lung Cancer (NSCLC): This is the most common type of lung cancer. Pralsetinib is being used to treat advanced or metastatic NSCLC with RET fusion.
- Medullary Thyroid Cancer (MTC): This is a rare type of thyroid cancer that starts in the C cells of the thyroid gland. Pralsetinib is being studied for use in MTC with RET mutations.
- Other RET-altered Solid Tumors: Pralsetinib is also being investigated for use in other types of solid tumors that have RET fusions or mutations.
How Pralsetinib is Administered
Pralsetinib is taken orally (by mouth) in the form of capsules. The typical dose is 400 mg once daily. However, the exact dosage may vary depending on the specific condition being treated and the patient's individual factors. It's important to take pralsetinib exactly as prescribed by your healthcare provider.
Ongoing Clinical Trials
Several clinical trials are currently underway to further study the effectiveness and safety of pralsetinib in various settings:
- First-line Treatment: A study is comparing pralsetinib to standard chemotherapy as the first treatment for patients with advanced RET fusion-positive NSCLC.
- Medullary Thyroid Cancer: A trial is evaluating pralsetinib compared to standard treatments (cabozantinib or vandetanib) in patients with RET-mutated MTC.
- Neoadjuvant Treatment: Pralsetinib is being studied as a treatment before surgery (neoadjuvant therapy) in patients with early-stage NSCLC that has RET fusions.
- Basket Trial: A large study called TAPISTRY is investigating pralsetinib and other targeted therapies in patients with various types of solid tumors that have specific genetic alterations, including RET fusions.
Potential Side Effects
As with all medications, pralsetinib can cause side effects. Common side effects may include:
- Fatigue
- Constipation
- Muscle and joint pain
- High blood pressure
- Decreased white blood cell counts
It's important to discuss potential side effects with your healthcare provider. They can provide more detailed information and help manage any side effects that may occur.